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UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

Form 10-Q


☒	QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934

For the quarterly period ended September 30, 2025 or


☐	TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934

For the transition period from to

Commission File Number: 001-36829

Rocket Pharmaceuticals, Inc.

(Exact name of registrant as specified in its charter)


Delaware		04-3475813
(State or other jurisdiction of incorporation or organization)		(I.R.S. Employer Identification No.)

9 Cedarbrook Drive, Cranbury, NJ		08512
(Address of principal executive office)		(Zip Code)

(609) 659-8001

(Registrant’s telephone number, including area code)

Securities registered pursuant to Section 12(b) of the Act:


Title of each class	Trading Symbol(s)	Name of each exchange on which registered
Common Stock, $0.01 par value per share	RCKT	Nasdaq Global Market

Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days. Yes ☒ No ☐

Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T (§ 232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files). Yes ☒ No ☐

Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, a smaller reporting company, or an emerging growth company. See the definitions of “large accelerated filer,” “accelerated filer,” “smaller reporting company” and “emerging growth company” in Rule 12b-2 of the Exchange Act.


Large accelerated filer ☒		Accelerated filer ☐
Non-accelerated filer ☐		Smaller reporting company ☐
		Emerging growth company ☐

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐

Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act). Yes ☐ No ☒

As of October 31, 2025, there were 108,222,228 shares of common stock, $0.01 par value per share, outstanding.

Table of Contents


		Page
	Summary of Abbreviated Terms	3
	Cautionary Statement Regarding Forward-Looking Statements	4
	PART I - FINANCIAL INFORMATION	6
Item 1.	Financial Statements	6
	Consolidated Balance Sheets as of September 30, 2025 (unaudited) and December 31, 2024	6
	Consolidated Statements of Operations for the three and nine months ended September 30, 2025 and 2024 (unaudited)	7
	Consolidated Statements of Comprehensive Loss for the three and nine months ended September 30, 2025 and 2024 (unaudited)	8
	Consolidated Statements of Stockholders’ Equity for the three and nine months ended September 30, 2025 and 2024 (unaudited)	9
	Consolidated Statements of Cash Flows for the nine months ended September 30, 2025 and 2024 (unaudited)	10
	Notes to Consolidated Financial Statements (unaudited)	11
Item 2.	Management’s Discussion and Analysis of Financial Condition and Results of Operations	26
Item 3.	Quantitative and Qualitative Disclosures About Market Risk	42
Item 4.	Controls and Procedures	43
	PART II - OTHER INFORMATION	44
Item 1.	Legal Proceedings	44
Item 1A.	Risk Factors	44
Item 2.	Unregistered Sales of Equity Securities and Use of Proceeds	45
Item 3.	Defaults Upon Senior Securities	45
Item 4.	Mine Safety Disclosures	45
Item 5.	Other Information	45
Item 6.	Exhibits	46
Signatures		47

Table of Contents

Summary of Abbreviated Terms

Rocket Pharmaceuticals, Inc. may be referred to as Rocket, the Company, we, our or us, in this Quarterly Report, unless the context otherwise indicates. Throughout this Quarterly Report, we have used terms which are defined below:


AAV	Adeno-associated virus	IND	Investigational New Drug application
ACM	Arrhythmogenic cardiomyopathy	IPR&D	In process research and development
ASC	Accounting Standard Codification	KCCQ	Kansas City Cardiovascular Questionnaire
ASGCT	American Society of Gene & Cell Therapy	LAD-I	Leukocyte Adhesion Deficiency-I
ATMP	Advanced Therapy Medical Product	LAMP2	Lysosome-associated membrane protein 2
BAG3	BCL2-associated athanogene 3	LV	Lentiviral vector
BAG3-DCM	BCL2-associated athanogene 3 mutations associated with dilated cardiomyopathy	MAA	Marketing Authorization Application
BLA	Biologics License Application	NYHA	New York Heart Association
BNP	Brain natriuretic peptide	Offering	Equity offering of shares of common stock, which closed December 12, 2024
cGMP	Current Good Manufacturing Practice	PDUFA	Prescription Drug User Fee Act
CIRM	California Institute for Regenerative Medicine	PKD	Pyruvate Kinase Deficiency
CMC	Chemistry Manufacturing Controls	PKP2	Plakophilin-2
CODM	Chief Operating Decision Maker	PKP2-ACM	Plakophilin-2 Arrhythmogenic Cardiomyopathy
CRL	Complete Response Letter	PSU	Performance Stock Unit
Cowen	Cowen and Company, LLC	PRIME	Priority Medicines
CTIS	Clinical Trials Information Systems	Private Placement	Issuance of pre-funded warrants on December 12, 2024
DCM	Dilated cardiomyopathy	R&D	Research and development
DD	Danon Disease	RBC	Red blood cell
DNA	Deoxyribonucleic acid	Renovacor	Renovacor, Inc. acquired on December 1, 2022
ECG	Electrocardiogram	RMAT	Regenerative Medicine Advanced Therapy
EMA	European Medicines Agency	RSU	Restricted stock unit
ESB Lease Agreement	Office Lease agreement for office space in the Empire State Building in New York City	RTW	RTW Investments, L.P
EU	European Union	SAE	Serious Adverse Event
FA	Fanconi Anemia	SCD	Sudden cardiac death
FDA	U.S. Food and Drug Administration	SEC	Securities and Exchange Commission
GMP	Good Manufacturing Practice	Stanford	Center for Definitive and Curative Medicine at Stanford University School of Medicine
HF	Heart Failure	UCLA	University of California, Los Angeles
HNJ	Hospital Infantil de Niño Jesús	U.S.	United States
HSCT	Hematopoietic stem cell transplant	U.S. GAAP	U.S. Generally Accepted Accounting Principles
ICD	Implantable cardiac defibrillator

Table of Contents

Cautionary Statement Regarding Forward-Looking Statements

This Quarterly Report on Form 10-Q for the quarter ended September 30, 2025 contains forward-looking statements that involve risks and uncertainties, as well as assumptions that, if they do not materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. All statements other than statements of historical facts contained in this Quarterly Report on Form 10-Q are forward-looking statements. In some cases, you can identify forward-looking statements by words such as “aim,” “anticipate,” “believe,” “can,” “contemplate,” “continue,” “could,” “design,” “develop,” “estimate,” “expect,” “expand,” “future,” “hope,” “intend,” “likely,” “may,” “plan,” “potential,” “predict,” “project,” “pursue,” “seek,” “should,” “strategy,” “target,” “will,” “would,” or the negative of these words or other comparable terminology. These forward-looking statements include, but are not limited to, statements about:

•

our ability to meet our anticipated milestones for our various drug candidates with respect to the initiation and timing of clinical studies;

•

federal, state, and non-U.S. regulatory requirements, including regulation of our current or any other future product candidates by the FDA;

•

the timing of and our ability to submit regulatory filings with the FDA and to obtain and maintain FDA or other regulatory authority approval of, or other action with respect to, our product candidates;

•

our competitors’ activities, including decisions as to the timing of competing product launches, pricing, and discounting;

•

whether safety and efficacy results of our clinical trials and other required tests for approval of our product candidates provide data to warrant progression of clinical trials, potential regulatory approval, or further development of any of our product candidates;

•

our ability to develop, acquire and advance product candidates into, enroll a sufficient number of patients into, and successfully complete, clinical studies, and our ability to apply for and obtain regulatory approval for such product candidates, within currently anticipated timeframes, or at all;

•

our ability to establish key collaborations and vendor relationships for our product candidates and any other future product candidates;

•

our ability to develop our sales and marketing capabilities or enter into agreements with third parties to sell and market any of our product candidates;

•

our ability to obtain additional funding to conduct our planned research and development efforts;

•

our ability to acquire additional businesses, form strategic alliances or create joint ventures and our ability to realize the benefit of such acquisitions, alliances, or joint ventures;

•

our ability to successfully develop and commercialize any technology that we may in-license or products we may acquire;

•

the development of our direct manufacturing capabilities for our AAV programs;

•

our ability to expand our pipeline to target additional indications that are compatible with our gene therapy technologies;

•

our ability to achieve the expected benefits of our portfolio prioritization and strategic restructuring and our estimates related to the costs and timing of implementing such initiative;

•

our ability to successfully operate in non-U.S. jurisdictions in which we currently or in the future do business, including compliance with applicable regulatory requirements and laws;

•

our ability to obtain and enforce patents to protect our product candidates, and our ability to successfully defend ourselves against unforeseen third-party infringement claims;

•

anticipated trends and challenges in our business and the markets in which we operate;

•

our estimates regarding our capital requirements; and

•

our ability to obtain additional financing and raise capital as necessary to fund operations or pursue business opportunities.

We caution you that the foregoing list may not contain all of the forward-looking statements made in this Quarterly Report on Form 10-Q.

Any forward-looking statements in this Quarterly Report on Form 10-Q reflect our current views with respect to future events or to our future financial performance and involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance, or achievements to be materially different from any future results, performance or achievements expressed or implied by these forward-looking statements. We have included important factors in the cautionary statements included in this Quarterly Report on Form 10-Q, particularly in the “Risk Factors” section incorporated by reference from our Annual Report for the year ended December 31, 2024, on Form 10-K, that could cause actual results or events to differ materially from the forward-looking statements that we make. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Our forward-looking statements do not reflect the potential impact of any future acquisitions, mergers, dispositions, joint ventures, or investments we may make or enter into.

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You should read this Quarterly Report on Form 10-Q and the documents that we have filed as exhibits to this Quarterly Report on Form 10-Q completely and with the understanding that our actual future results, performance, or achievements may be materially different from what we expect. Except as required by law, we assume no obligation to update or revise these forward-looking statements for any reason, even if new information becomes available in the future.

This Quarterly Report on Form 10-Q also contains estimates, projections and other information concerning our industry, our business, and the markets for certain diseases, including data regarding the estimated size of those markets, and the incidence and prevalence of certain medical conditions. Information that is based on estimates, forecasts, projections, market research or similar methodologies is inherently subject to uncertainties and actual events, or circumstances may differ materially from events and circumstances reflected in this information. Unless otherwise expressly stated, we obtained this industry, business, market and other data from reports, research surveys, studies and similar data prepared by market research firms and other third parties, industry, medical and general publications, government data and similar sources. This Quarterly Report contains summaries of certain provisions contained in some of the documents described herein, but reference is made to the actual documents for complete information. All of the summaries are qualified in their entirety by the actual documents.

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PART I — FINANCIAL INFORMATION

Item 1. Financial Statements

Rocket Pharmaceuticals, Inc.

Consolidated Balance Sheets

($ in thousands, except shares and per share amounts)


	September 30, 2025			December 31, 2024
	(unaudited)
Assets
Current assets:
Cash and cash equivalents	$	75,948		$	163,635
Investments		146,810			208,701
Prepaid expenses and other current assets		4,922			5,847
Total current assets		227,680			378,183
Property and equipment, net		29,768			36,786
Goodwill		39,154			39,154
Intangible assets		25,150			25,150
Restricted cash		1,365			1,362
Deposits		515			529
Operating lease right-of-use assets, net		3,653			4,173
Finance lease right-of-use asset, net		40,748			42,363
Total assets	$	368,033		$	527,700
Liabilities and stockholders' equity
Current liabilities:
Accounts payable and accrued expenses	$	28,258		$	37,827
Operating lease liabilities, current		1,015			1,001
Finance lease liability, current		1,897			1,856
Total current liabilities		31,170			40,684
Operating lease liabilities, non-current		2,758			3,258
Finance lease liability, non-current		19,375			19,383
Other liabilities		1,061			1,141
Total liabilities		54,364			64,466
Commitments and contingencies (Note 13)

Stockholders' equity:
Preferred stock, $0.01 par value, authorized 5,000,000 shares:
Series A convertible preferred stock; 300,000 shares designated; 0 shares issued and outstanding		-			-
Series B convertible preferred stock; 300,000 shares designated; 0 shares issued and outstanding		-			-
Common stock, $0.01 par value, 180,000,000 shares authorized; 108,208,643 and 106,453,818 shares issued and outstanding as of September 30, 2025, and December 31, 2024, respectively		1,082			1,065
Additional paid-in capital		1,711,297			1,680,219
Accumulated other comprehensive (loss) income		(9	)		66
Accumulated deficit		(1,398,701	)		(1,218,116	)
Total stockholders’ equity		313,669			463,234
Total liabilities and stockholders’ equity	$	368,033		$	527,700

The accompanying notes are an integral part of these consolidated financial statements.

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Rocket Pharmaceuticals, Inc.

Consolidated Statements of Operations

($ in thousands, except shares and per share amounts)

(unaudited)


	Three Months Ended September 30,						Nine Months Ended September 30,
	2025			2024			2025			2024
Revenue	$	-		$	-		$	-		$	-

Operating expenses:
Research and development		34,068			42,315			112,668			133,887
General and administrative		18,351			27,109			71,817			76,624
Restructuring		(172	)		-			3,299			-
Total operating expenses		52,247			69,424			187,784			210,511
Loss from operations		(52,247	)		(69,424	)		(187,784	)		(210,511	)
Interest expense		(473	)		(471	)		(1,418	)		(1,413	)
Interest and other income, net		709			1,327			2,528			6,650
Accretion of discount on investments, net		1,679			1,849			6,089			6,855
Net loss	$	(50,332	)	$	(66,719	)	$	(180,585	)	$	(198,419	)
Net loss per share - basic and diluted	$	(0.45	)	$	(0.71	)	$	(1.63	)	$	(2.11	)
Weighted-average common shares outstanding - basic and diluted		111,571,136			94,158,491			110,900,161			93,893,729

The accompanying notes are an integral part of these consolidated financial statements.

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Rocket Pharmaceuticals, Inc.

Consolidated Statements of Comprehensive Loss

($ in thousands)

(unaudited)


	Three Months Ended September 30,						Nine Months Ended September 30,
	2025			2024			2025			2024
Net loss	$	(50,332	)	$	(66,719	)	$	(180,585	)	$	(198,419	)
Other comprehensive loss:
Net unrealized gain (loss) on investments		98			431			(75	)		(106	)
Total comprehensive loss	$	(50,234	)	$	(66,288	)	$	(180,660	)	$	(198,525	)

The accompanying notes are an integral part of these consolidated financial statements.

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Rocket Pharmaceuticals, Inc.

Consolidated Statements of Stockholders’ Equity

($ in thousands except share amounts)

(unaudited)


									Accumulated
						Additional			Other						Total
	Common Stock					Paid-In			Comprehensive			Accumulated			Stockholders'
	Shares			Amount		Capital			Income/(Loss)			Deficit			Equity
Balance at December 31, 2024		106,453,818		$	1,065	$	1,680,219		$	66		$	(1,218,116	)	$	463,234
Issuance of common stock pursuant to vesting of restricted stock units		300,068			3		(3	)		-			-			-
Unrealized comprehensive loss on investments		-			-		-			(93	)		-			(93	)
Stock-based compensation		-			-		10,331			-			-			10,331
Net loss		-			-		-			-			(61,334	)		(61,334	)
Balance at March 31, 2025		106,753,886			1,068		1,690,547			(27	)		(1,279,450	)		412,138
Issuance of common stock pursuant to exercise of stock options		952,313			9		(9	)		-			-			-
Issuance of common stock pursuant to vesting of restricted stock units		178,221			2		(2	)		-			-			-
Unrealized comprehensive loss on investments		-			-		-			(80	)		-			(80	)
Stock-based compensation		-			-		10,857			-			-			10,857
Return of related party short-swing profits		-			-		215			-			-			215
Net loss		-			-		-			-			(68,919	)		(68,919	)
Balance at June 30, 2025		107,884,420			1,079		1,701,608			(107	)		(1,348,369	)		354,211
Vesting of restricted stock units		347,031			3		(3	)		-			-			-
Repurchase of restricted stock units to satisfy tax withholding		(22,808	)		-		(67	)		-			-			(67	)
Unrealized comprehensive gain on investments		-			-		-			98			-			98
Stock-based compensation		-			-		9,759			-			-			9,759
Net loss		-			-		-			-			(50,332	)		(50,332	)
Balance at September 30, 2025		108,208,643		$	1,082	$	1,711,297		$	(9	)	$	(1,398,701	)	$	313,669


								Accumulated
					Additional			Other						Total
	Common Stock				Paid-In			Comprehensive			Accumulated			Stockholders'
	Shares		Amount		Capital			Income/(Loss)			Deficit			Equity
Balance at December 31, 2023		90,282,267	$	903	$	1,450,722		$	319		$	(959,370	)	$	492,574
Issuance of common stock pursuant to exercise of stock options		73,745		-		1,184			-			-			1,184
Issuance of common stock pursuant to vesting of restricted stock units		290,578		3		(3	)		-			-			-
Unrealized comprehensive loss on investments		-		-		-			(454	)		-			(454	)
Stock-based compensation		-		-		10,252			-			-			10,252
Net loss		-		-		-			-			(62,054	)		(62,054	)
Balance at March 31, 2024		90,646,590		906		1,462,155			(135	)		(1,021,424	)		441,502
Issuance of common stock pursuant to exercise of stock options		159,355		2		1,528			-			-			1,530
Issuance of common stock pursuant to vesting of restricted stock units		150,668		2		(2	)		-			-			-
Unrealized comprehensive loss on investments		-		-		-			(83	)		-			(83	)
Stock-based compensation		-		-		11,332			-			-			11,332
Net loss		-		-		-			-			(69,646	)		(69,646	)
Balance at June 30, 2024		90,956,613		910		1,475,013			(218	)		(1,091,070	)		384,635
Issuance of common stock pursuant to exercise of stock options		13,108				176									176
Issuance of common stock pursuant to vesting of restricted stock units		146,971		1		(1	)								-
Unrealized comprehensive gain on investments									431						431
Stock-based compensation						11,248									11,248
Net loss												(66,719	)		(66,719	)
Balance at September 30, 2024		91,116,692	$	911	$	1,486,436		$	213		$	(1,157,789	)	$	329,771

The accompanying notes are an integral part of these consolidated financial statements.

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Rocket Pharmaceuticals, Inc.

Consolidated Statements of Cash Flows

($ in thousands)

(unaudited)


	Nine Months Ended September 30,
	2025			2024
Operating activities:
Net loss	$	(180,585	)	$	(198,419	)
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation and amortization of property and equipment		7,193			5,399
Amortization of finance lease right of use asset		1,615			1,615
Stock-based compensation		30,947			32,832
Accretion of discount on investments, net		(5,886	)		(6,845	)
Change in fair value of warrant liabilities		-			(1,875	)
Changes in operating assets and liabilities:
Prepaid expenses and other assets		939			(1,015	)
Accounts payable and accrued expenses		(9,384	)		5,293
Operating lease liabilities and right of use assets, net		34			72
Finance lease liability		33			80
Other liabilities		(80	)		81
Net cash used in operating activities		(155,174	)		(162,782	)
Investing activities:
Purchases of investments		(222,058	)		(121,841	)
Proceeds from maturities of investments		289,760			296,971
Purchases of property and equipment		(360	)		(5,553	)
Net cash provided by investing activities		67,342			169,577
Financing activities:
Issuance of common stock pursuant to exercise of stock options		-			2,890
Return of related party short-swing profits		215			-
Repurchase of restricted stock units to satisfy tax withholding		(67	)		-
Net cash provided by financing activities		148			2,890
Net change in cash, cash equivalents and restricted cash		(87,684	)		9,685
Cash, cash equivalents and restricted cash at beginning of period		164,997			57,276
Cash, cash equivalents and restricted cash at end of period	$	77,313		$	66,961

Supplemental disclosure of non-cash financing and investing activities:
Accrued purchases of property and equipment, ending balance	$	-		$	265
Operating lease liabilities	$	-		$	1,134
Operating lease right of use assets	$	-		$	1,134
Net unrealized loss on investments	$	(75	)	$	(106	)

The accompanying notes are an integral part of these consolidated financial statements.

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Rocket Pharmaceuticals, Inc.

Notes to Consolidated Financial Statements

($ in thousands, except shares and per share data) (Unaudited)

Nature of Business

Rocket Pharmaceuticals, Inc. (the “Company”) is a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare disorders. Rocket’s innovative multi-platform approach allows the Company to design the optimal gene therapy for each indication, creating potentially transformative options that enable people living with devastating rare diseases to experience long and full lives.

Rocket’s in vivo adeno-associated viral (AAV) vector-based cardiovascular portfolio includes a late-stage clinical program for Danon disease, a devastating heart failure condition resulting in thickening of the heart, and early-stage clinical programs for PKP2-arrhythmogenic cardiomyopathy (PKP2-ACM), a life-threatening heart failure disease causing ventricular arrhythmias and sudden cardiac death, and BAG3-associated dilated cardiomyopathy (BAG3-DCM), a heart failure condition that causes enlarged ventricles.

Rocket’s ex vivo lentiviral (LV) vector-based hematology portfolio consists of late-stage programs for Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal; Fanconi Anemia (FA), a difficult-to-treat genetic disease that leads to bone marrow failure and potentially cancer; and Pyruvate Kinase Deficiency (PKD), a monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia.

Risks and Liquidity

The Company has not generated any revenue and has incurred losses since inception. Operations of the Company are subject to certain risks and uncertainties, including, among others, uncertainty of drug candidate development, technological uncertainty, uncertainty regarding patents and proprietary rights, having no commercial manufacturing experience, marketing or sales capability or experience, dependency on key personnel, compliance with government regulations and the need to obtain additional financing. Drug candidates currently under development will require significant additional research and development efforts, including extensive preclinical and clinical testing and regulatory approval, prior to commercialization. These efforts require significant amounts of additional capital, adequate personnel infrastructure, and extensive compliance-reporting capabilities.

The Company’s product candidates are in the development and clinical stage. There can be no assurance that the Company’s research and development will be successfully completed, that adequate protection for the Company’s intellectual property will be obtained, that any products developed will obtain necessary government approval or that any approved products will be commercially viable. Even if the Company’s product development efforts are successful, it is uncertain when, if ever, the Company will generate significant revenue from product sales. The Company operates in an environment of rapid change in technology and substantial competition from pharmaceutical and biotechnology companies.

The Company’s consolidated financial statements have been prepared on the basis of continuity of operations, realization of assets and the satisfaction of liabilities in the ordinary course of business. The Company has experienced negative cash flows from operations and had an accumulated deficit of $1.40 billion as of September 30, 2025. As of September 30, 2025, the Company had $222.8 million of cash, cash equivalents and investments. The Company expects that in accordance with the current operating plan, which reflects a strategic corporate reorganization and reprioritization announced in July 2025, such resources will be sufficient to fund the Company’s operating expenses and capital expenditure requirements into the second quarter of 2027.

In the longer term, the future viability of the Company is dependent on its ability to generate cash from operating activities or to raise additional capital to finance its operations. The Company expects to continue to generate operating losses for the foreseeable future and to finance its future cash needs through, but not limited to, one or a combination of equity offerings, debt financings, collaborations, strategic partnerships and alliances or licensing arrangements. If the Company is unable to obtain funding, the Company would be forced to delay, reduce or eliminate some or all of its research and development programs, preclinical and clinical testing or commercialization efforts, which could adversely affect its business prospects. The Company’s failure to raise capital as and when needed could have a negative impact on its financial condition and ability to pursue its business strategies.

In May 2025, two patients participating in the Phase 2 pivotal study of RP-A501 each experienced an unexpected SAE. Rocket voluntarily paused further Phase 2 study dosing in the U.S. and E.U., and the FDA placed a clinical hold on the trial to allow for further evaluation. On August 20, 2025, the Company announced that the FDA lifted the clinical hold on the trial after the Company satisfactorily addressed issues outlined in the clinical hold.

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In July 2025, the Company implemented a strategic corporate reorganization designed to align its resources with its highest-priority programs, namely, the AAV-based gene therapy platform focused on cardiovascular diseases. As part of this effort, the Company reduced its workforce by approximately 30%.

While the reorganization is intended to streamline operations, improve capital efficiency, and extend the Company’s cash runway, there can be no assurance that the anticipated benefits such as cost savings, improved focus, or operational efficiency will be realized to the extent or within the timeframe currently expected. The reduction in force and related organizational changes may also result in near-term disruption to the Company’s operations, loss of institutional knowledge, or diminished employee morale, which could adversely impact productivity and the Company’s ability to execute on key initiatives. In addition, the Company may incur additional or unforeseen costs associated with the reorganization, including severance, outplacement, legal, and consulting expenses.

The Company may also face challenges retaining key personnel and maintaining continuity across teams, which could impair our ability to advance clinical programs, meet regulatory milestones, or pursue long-term strategic objectives. Potential litigation or other employee-related claims arising from the workforce reduction could divert management attention and further increase costs. Any of these factors could have a material adverse effect on our business, operating results, and financial condition.

Basis of Presentation, Principles of Consolidation and Summary of Significant Accounting Policies

Basis of Presentation

The accompanying unaudited interim consolidated financial statements should be read in conjunction with the Company’s consolidated financial statements for the year ended December 31, 2024 included in the Annual Report on Form 10-K filed with the Securities and Exchange Commission on February 27, 2025. The unaudited interim consolidated financial statements have been prepared on the same basis as the audited annual financial statements and, in the opinion of management, reflect all adjustments, which include only normal recurring adjustments, necessary for the fair statement of the Company’s consolidated financial position as of September 30, 2025 and the results of its operations and its cash flows for the nine months ended September 30, 2025. The financial data and other information disclosed in these consolidated notes related to the three and nine months ended September 30, 2025 and 2024 are unaudited. The results for the three and nine months ended September 30, 2025 are not necessarily indicative of results to be expected for the year ending December 31, 2025 and any other interim periods or any future year or period.

Significant Accounting Policies

The significant accounting policies used in the preparation of these consolidated financial statements for the three and nine months ended September 30, 2025 are consistent with those disclosed in Note 3 to the consolidated financial statements in the 2024 Form 10-K with most significant policies also being listed here.

Principles of Consolidation

The consolidated financial statements represent the consolidation of the accounts of the Company and its subsidiaries in conformity with U.S. GAAP. All intercompany accounts have been eliminated in consolidation.

Use of Estimates

The preparation of the consolidated financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and disclosure of contingent assets and liabilities at the date of the financial statements and the reported amounts of expenses during the reporting period. Significant estimates and assumptions reflected in these consolidated financial statements include but are not limited to goodwill and intangible asset impairments, the accrual of R&D expenses, the valuation of equity transactions, and stock-based awards. Changes in estimates and assumptions are reflected in reported results in the period in which they become known. Actual results could differ from those estimates.

Cash, Cash Equivalents and Restricted Cash

Cash, cash equivalents and restricted cash consists of bank deposits, certificates of deposit and money market accounts with financial institutions. Cash equivalents are carried at cost, which approximates fair value due to their short-term nature and which the Company believes do not have a material exposure to credit risk. The Company considers all highly liquid investments with maturities of three months or less from the date of purchase to be cash equivalents. The Company’s cash and cash equivalent accounts, at times, may exceed federally insured limits. The Company has not experienced any losses in such accounts.

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Restricted cash consists of deposits collateralizing letters of credit issued by a bank in connection with the Company’s operating leases (see Note 12 “Leases” for additional disclosures) and a deposit collateralizing a letter of credit issued by a bank supporting the Company’s corporate credit cards. Cash, cash equivalents and restricted cash consist of the following:


	September 30, 2025		December 31, 2024
Cash and cash equivalents	$	75,948	$	163,635
Restricted cash		1,365		1,362
Total cash, cash equivalents and restricted cash	$	77,313	$	164,997

Concentrations of credit risk and off-balance sheet risk

Financial instruments that subject the Company to credit risk primarily consist of cash and cash equivalents and available-for-sale securities. The Company maintains its cash and cash equivalent balances with high quality financial institutions and, consequently, the Company believes that such funds are subject to minimal credit risk. The Company’s marketable securities consist of U.S. Treasury Securities. The Company’s investment policy limits the amounts the Company may invest in any one type of investment and requires all investments held by the Company to be at least AA-/Aa3 rated, thereby reducing credit risk exposure.

Investments

Investments consist of U.S. Treasury Securities. Management determines the appropriate classification of these securities at the time they are acquired and evaluates the appropriateness of such classifications at each balance sheet date. The Company classifies its investments as available-for-sale pursuant to ASC 320, Investments-Debt and Equity Securities. Investments are recorded at fair value, with unrealized gains and losses included as a component of accumulated other comprehensive income (loss) in stockholders’ equity and a component of total comprehensive loss in the consolidated statements of comprehensive loss, until realized. Realized gains and losses are included in investment income on a specific-identification basis. The Company estimates expected credit losses for investments when unrealized losses exist. Unrealized losses that are credit related are recognized in the Company’s Consolidated Statement of Operations and unrealized losses that are not credit related are recognized in accumulated other comprehensive income (loss). For the three and nine months ended September 30, 2025 and 2024, there were no unrealized losses that were credit related. For the three and nine months ended September 30, 2025, there were net unrealized gain on investments of $0.1 million and net unrealized loss on investment of $0.1 million, respectively. For the three and nine months ended September 30, 2024, there were net unrealized gain on investments of $0.4 million and net unrealized loss on investment of $0.1 million, respectively.

Intangible Assets

Intangible assets consisted of an indefinite lived intangible IPR&D asset. Intangible assets related to IPR&D projects are considered to be indefinite-lived until the completion or abandonment of the associated R&D efforts. If and when development is complete, which generally occurs if and when regulatory approval to market a product is obtained, the associated assets would be deemed finite-lived and would then be amortized based on their respective estimated useful lives at that point in time. IPR&D intangible assets which are determined to have had a decrease in their fair value are adjusted downward and an expense is recognized in R&D expenses in the Consolidated Statements of Operations. These IPR&D intangible assets are tested at least annually or when a triggering event occurs that could indicate a potential impairment based on indicators including progress of R&D activities, changes in projected development of assets, and changes in regulatory environment and future commercial markets. If a triggering event occurs that would indicate a potential impairment, the Company will perform a quantitative analysis to determine whether it is more likely than not that the fair value is below the carrying amount.

Goodwill

Goodwill is tested for impairment annually as of December 31, or more frequently when events or changes in circumstances indicate that the asset might be impaired.

In May 2025, two patients participating in the Phase 2 pivotal study of RP-A501 each experienced an unexpected SAE after which the FDA placed a clinical hold on the trial to allow for further evaluation. In response to the unexpected SAEs, the Company performed a quantitative assessment of its goodwill and determined that it is more likely than not that the fair value of the Company exceeds the carrying value. As a result, the Company has determined that there was no goodwill impairment as of the date of the event or for the three and nine months ended September 30, 2025. The clinical hold on the trial was subsequently lifted by the FDA after the Company satisfactorily addressed issues outlined in the clinical hold.

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Fair Value Measurements

The Company is required to disclose information on all assets and liabilities reported at fair value that enables an assessment of the inputs used in determining the reported fair values. ASC 820, Fair Value Measurements and Disclosures, establishes a hierarchy of inputs used when available. Observable inputs are inputs that market participants would use in pricing the asset or liability based on market data obtained from sources independent of the Company. Unobservable inputs are inputs that reflect the Company’s assumptions about the inputs that market participants would use in pricing the asset or liability and are developed based on the best information available in the circumstances. The fair value hierarchy applies only to the valuation inputs used in determining the reported fair value of the investments and is not a measure of the investment credit quality. The three levels of the fair value hierarchy are described below:

•

Level 1 - Valuations based on unadjusted quoted prices in active markets for identical assets or liabilities that the Company has the ability to access at the measurement date.

•

Level 2 - Valuations based on quoted prices for similar assets or liabilities in markets that are not active or for which all significant inputs are observable, either directly or indirectly.

•

Level 3 - Valuations that require inputs that reflect the Company’s own assumptions that are both significant to the fair value measurement and unobservable.

To the extent that valuation is based on models or inputs that are less observable or unobservable in the market, the determination of fair value requires more judgment. Accordingly, the degree of judgment exercised by the Company in determining fair value is greatest for instruments categorized in Level 3. A financial instrument’s level within the fair value hierarchy is based on the lowest level of any input that is significant to the fair value measurement. The fair value of the Company’s financial instruments, including cash and cash equivalents, restricted cash, deposits, accounts payable and accrued expenses approximate their respective carrying values due to the short-term nature of most of these instruments.

Warrants

The Company accounts for stock warrants as either equity instruments, liabilities or derivative liabilities in accordance with ASC 480, Distinguishing Liabilities from Equity and/or ASC 815, Derivatives and Hedging, depending on the specific terms of the warrant agreement. Liability-classified warrants are recorded at their estimated fair values at each reporting period until they are exercised, terminated, reclassified or otherwise settled. Changes in the estimated fair value of liability-classified warrants are included in interest and other income in the Company’s Consolidated Statement of Operations. Warrants classified as equity instruments are recorded within additional paid-in capital at the time of issuance and are not subject to remeasurement.

Stock-Based Compensation

The Company issues stock-based awards to employees and non-employees, generally in the form of stock options, RSUs and PSUs.

The Company measures the compensation expense of employee and non-employee services received in exchange for an award of equity instruments based on the fair value of the award on the grant date. The cost of a stock option or RSU is recognized over the requisite service period of the award on a straight-line basis with forfeitures recognized as they occur. The vesting condition for PSUs is performance based and the cost of a PSU is recognized when it is likely that the performance goal associated with the PSU will be achieved and the award will vest.

The fair value of options on the date of grant is calculated using the Black-Scholes option pricing model based on key assumptions such as expected volatility and expected term.

The Company classifies stock-based compensation expense in its Consolidated Statements of Operations in the same manner in which the award recipient’s payroll costs and services are classified or in which the award recipient’s service payments are classified.

Segment Reporting

Operating segments are defined as components of an enterprise about which separate discrete information is available for evaluation by the chief operating decision maker, or decision-making group, in deciding how to allocate resources and in assessing performance. The Company views its operations and manages its business in one operating segment. The Company’s CODM is its Chief Executive Officer and the senior leadership team. The CODM manages the Company’s operations on an integrated basis for the purpose of allocating resources. When evaluating the Company’s financial performance, the CODM regularly reviews total expenses and expenses by significant areas to make decisions on a company wide basis. Included in these expenses are R&D expenses by program.

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Income Taxes

On July 4, 2025, the One Big Beautiful Bill Act was enacted into law with changes to U.S. tax law that will be applicable to the Company beginning in 2025. These changes include provisions allowing accelerated tax deductions for qualified property and research expenditures. We are in the process of evaluating the impact of the One Big Beautiful Bill Act on our Consolidated Financial Statements.

Recent Accounting Pronouncements

Accounting Pronouncements Not Adopted as of September 30, 2025

ASU 2023-09: Income Taxes Topic 740 - Improvements to Income Tax Disclosures. This update standardizes categories for the effective tax rate reconciliation, requires disaggregation of income taxes and additional income tax-related disclosures. This update is required to be effective for the Company for fiscal periods beginning after December 15, 2024. The Company is evaluating the effect that ASU 2023-09 will have on its financial statements and disclosures.

ASU 2024-03: Expense Disaggregation Disclosures. This update requires disaggregated disclosure of income statement expenses. This update will be effective for the Company for fiscal years beginning after December 15, 2027. Early adoption is permitted. The Company is evaluating the effect that ASU 2024-03 will have on its financial statements and disclosures.

Fair Value of Financial Instruments

Items measured at fair value on a recurring basis are the Company’s investments and warrant liability. The following table sets forth the Company’s financial investments that were measured at fair value on a recurring basis by level within the fair value hierarchy:


	Fair Value Measurements as of September 30, 2025, Using:
	Level 1		Level 2		Level 3		Total
Assets:
Cash equivalents:
Money market mutual funds	$	50,286	$	-	$	-	$	50,286
U.S. Treasury securities		-		4,999		-		4,999
		50,286		4,999		-		55,285

Investments:
U.S. Treasury securities		-		146,810		-		146,810
		-		146,810		-		146,810

Total assets	$	50,286	$	151,809	$	-	$	202,095

	Fair Value Measurements as of December 31, 2024, Using:
	Level 1		Level 2		Level 3		Total
Assets:
Cash equivalents:
Money market mutual funds	$	139,948	$	-	$	-	$	139,948
U.S. Treasury securities		-		7,453		-		7,453
		139,948		7,453		-		147,401

Investments:
U.S. Treasury securities		-		208,701		-		208,701
		-		208,701		-		208,701

Total assets	$	139,948	$	216,154	$	-	$	356,102

The Company classifies its money market mutual funds as Level 1 assets under the fair value hierarchy, as these assets have been valued using quoted market prices in active markets without any valuation adjustment. The Company classifies its U.S. Treasury Securities as Level 2 assets as these assets are not traded in an active market and have been valued through a third-party pricing service based on quoted prices for similar assets.

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The Company had a warrant liability, which expired on April 23, 2025. The warrant liability was recorded as part of other liabilities in the Consolidated Balance Sheets and measured at fair value on a recurring basis using unobservable inputs (Level 3). The warrant liability balance was approximately $0 on December 31, 2024.

Property and Equipment, Net

The Company’s property and equipment consisted of the following:


	September 30, 2025			December 31, 2024
Laboratory equipment	$	32,058		$	32,205
Machinery and equipment		12,133			12,857
Computer equipment		1,015			1,015
Furniture and fixtures		2,777			2,777
Leasehold improvements		7,327			7,282
Internal use software		1,903			1,903
		57,213			58,039
Less: accumulated depreciation and amortization		(27,445	)		(21,253	)
Total property and equipment, net	$	29,768		$	36,786

During the three and nine months ended September 30, 2025, the Company recognized $2.7 million and $7.2 million of depreciation and amortization expense, respectively. During the three and nine months ended September 30, 2024, the Company recognized $1.9 million and $5.4 million of depreciation and amortization expense, respectively.

Intangible Assets and Goodwill

The Company’s intangible assets consisted of an acquired IPR&D asset received in the acquisition of Renovacor. Intangible assets as of September 30, 2025, and December 31, 2024, are summarized as follows:


	September 30, 2025		December 31, 2024
Gross carrying value	$	25,150	$	25,150
Accumulated amortization		-		-
Total intangible assets	$	25,150	$	25,150

The carrying value of Goodwill as of September 30, 2025, and December 31, 2024, was $39.2 million.

Accounts Payable and Accrued Expenses

The Company’s accounts payable and accrued expenses consisted of the following:


	September 30, 2025		December 31, 2024
Research and development	$	9,577	$	16,768
Employee compensation		11,429		11,944
Property and equipment		-		185
Professional fees		1,819		7,305
Restructuring		2,760		-
Other		2,673		1,625
Total accounts payable and accrued expenses	$	28,258	$	37,827

Stockholders’ Equity

Public Offerings and Private Placements

On December 12, 2024, the Company completed the Offering of 15,180,000 shares of its common stock at a public offering price of $12.50 per share and a Private Placement of pre-funded warrants to purchase 400,000 shares of common stock at a price of $12.49 per pre-funded warrant. The gross proceeds from the Offering and Private Placement were approximately $194.7 million. The net proceeds of the Offering and Private Placement were approximately $182.5 million after reduction for offering costs, underwriting discounts and commissions, legal and other expenses.

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Stock-Based Compensation

Stock Option Valuation

The weighted average assumptions that the Company used in a Black-Scholes pricing model to determine the fair value of stock options granted to employees, non-employees and directors were as follows:


	Nine Months Ended September 30,
	2025			2024
Risk-free interest rate		4.19	%		4.85	%
Expected term (in years)		5.51			5.82
Expected volatility		75.33	%		73.70	%
Expected dividend yield		0.00	%		0.00	%
Exercise price	$	8.62		$	27.24
Fair value of common stock	$	8.62		$	27.24

The following table summarizes stock option activity for the nine months ended September 30, 2025:


				Weighted		Weighted
				Average		Average		Aggregate
	Number of			Exercise		Contractual		Intrinsic
	Shares			Price		Term (Years)		Value
Outstanding as of December 31, 2024		16,044,686		$	16.19		4.67	$	63,295
Granted		2,243,352			8.62		8.26
Exercised		(952,313	)		0.00				-
Cancelled or forfeited		(664,722	)		20.17
Outstanding as of September 30, 2025		16,671,003		$	15.94		4.18	$	9,935

Options vested and exercisable as of September 30, 2025		13,776,919		$	16.51		3.25	$	9,823
Options unvested as of September 30, 2025		2,894,084		$	13.20		8.61	$	112

The weighted average grant-date fair value per share of stock options granted during the nine months ended September 30, 2025, and 2024 was $5.15 and $18.34, respectively.

The total fair value of options vested during the nine months ended September 30, 2025, and 2024 was $24.0 million and $23.6 million, respectively.

Restricted Stock Units

The following table summarizes the Company’s RSU activity for the nine months ended September 30, 2025:


				Weighted Average
	Number of			Grant Date
	Shares			Fair Value
Unvested as of December 31, 2024		1,414,210		$	23.02
Granted		5,622,352			6.60
Vested(1)		(825,320	)		21.16
Forfeited		(709,618	)		12.32
Unvested as of September 30, 2025		5,501,624		$	7.90

(1) Common stock issued is net of 22,808 RSUs related to taxes.

The total fair value of RSUs vested during the nine months ended September 30, 2025, and 2024 was $17.5 million and $11.0 million, respectively.

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Performance Stock Units

The following table summarizes the Company’s PSU activity for the nine months ended September 30, 2025:


				Weighted Average
	Number of			Grant Date
	Shares			Fair Value
Unvested as of December 31, 2024		69,661		$	28.71
Granted		-			-
Vested		-			-
Forfeited		(52,246	)		28.71
Unvested as of September 30, 2025		17,415		$	28.71

PSU vesting and expense recognition is based on achievement of specific performance goals within certain time periods. PSU awards that are not achieved within specific time periods are forfeited. No performance goals were probable of achievement as of September 30, 2025, and the time periods for two of three performance goals at the beginning of the year expired during the nine months ended September 30, 2025.

Stock-Based Compensation Expense

Stock-based compensation expense recognized by award type was as follows:


	Three Months Ended September 30,				Nine Months Ended September 30,
	2025		2024		2025		2024
Stock options	$	4,419	$	7,005	$	15,780	$	21,105
Restricted stock units		5,340		4,243		15,167		11,727
Total stock-based compensation expense	$	9,759	$	11,248	$	30,947	$	32,832

Stock-based compensation expense by classification included within the Consolidated Statements of Operations and Comprehensive Loss was as follows:


	Three Months Ended September 30,				Nine Months Ended September 30,
	2025		2024		2025		2024
Research and development	$	4,752	$	4,789	$	13,961	$	14,311
General and administrative		5,007		6,459		16,986		18,521
Total stock-based compensation expense	$	9,759	$	11,248	$	30,947	$	32,832

As of September 30, 2025, the Company had an aggregate of $58.7 million of unrecognized stock-based compensation expense related to stock options, RSU and PSU grants. The stock options and RSU grants had an aggregate of $58.2 million of unrecognized stock-based compensation expense, which is expected to be recognized over a weighted average period of 1.87 years.

10.

Warrants

A summary of the warrants outstanding as of September 30, 2025, is as follows:


Exercise Price	Outstanding		Grant/Assumption Date	Expiration Date
$57.11		603,386	December 21, 2020	December 21, 2030
$33.63		301,291	August 9, 2021	August 9, 2031
$22.51		153,155	December 17, 2021	December 17, 2031
$22.51		153,155	December 17, 2021	December 17, 2031
$65.23		760,086	December 1, 2022	December 1, 2026
$0.01		3,126,955	September 15, 2023	N/A
$0.01		400,000	December 12, 2024	N/A
Total		5,498,028

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Warrants Issued in Public Offerings

The Company issued warrants to a related party during the years ended December 31, 2024, and 2023. For the years ended December 31, 2024, and 2023, the Company sold pre-funded warrants to purchase 400,000 and 3,126,955 shares of common stock, respectively with an exercise price of $0.01 per share (see Note 8 “Stockholders Equity”). The pre-funded warrants were acquired by funds affiliated with RTW (see Note 16 “Related Party Transactions”).

Assumed Renovacor Warrants

In conjunction with the acquisition of Renovacor, the Company assumed pre-acquisition public warrants that were converted into warrants to purchase 760,086 shares of Rocket Pharmaceuticals, Inc. common stock at an exercise price of $65.23 per share.

In conjunction with the acquisition of Renovacor, the Company assumed pre-acquisition private warrants that were converted into warrants to purchase 617,050 shares of Rocket Pharmaceuticals, Inc. common stock at an exercise price of $65.23 per share. The Company determined that the private warrants did not meet all of the criteria for equity classification. Accordingly, the Company classified these as a derivative liability in other liabilities in the Consolidated Balance Sheets. The Company measured the fair value of these warrants at the end of each reporting period and recognized changes in the fair value from the prior period in the Company’s operating results for the current period. These warrants expired on April 23, 2025.

11.

Net Loss Per Share

Basic and diluted net loss per share attributable to common stockholders was calculated as follows:


	Three Months Ended September 30,						Nine Months Ended September 30,
	2025			2024			2025			2024
Numerator:
Net loss attributable to common stockholders	$	(50,332	)	$	(66,719	)	$	(180,585	)	$	(198,419	)
Denominator:
Weighted-average common shares outstanding - basic and diluted		111,571,136			94,158,491			110,900,161			93,893,729
Net loss per share attributable to common stockholders - basic and diluted	$	(0.45	)	$	(0.71	)	$	(1.63	)	$	(2.11	)

For the three and nine months ended September 30, 2025, the Company included the 3,126,955 potential shares from the pre-funded warrants acquired by RTW in 2023 and the 400,000 potential shares from the pre-funded warrants acquired by RTW in 2024 in the basic weighted-average common shares outstanding as the warrants only require the holder to pay $0.01 per share upon exercise.

For the three and nine months ended September 30, 2024, the Company included the 3,126,955 potential shares from the pre-funded warrants acquired by RTW in 2023 in the basic weighted-average common shares outstanding as the warrants only require the holder to pay $0.01 per share upon exercise.

The Company excluded the following potential shares of common stock, presented based on amounts outstanding at each period end, from the computation of diluted net loss per share attributable to common stockholders for the periods indicated because including them would have had an anti-dilutive effect:


	Three and Nine Months Ended September 30,
	2025		2024
Warrants exercisable for common shares		1,971,073		2,588,123
Restricted stock units		5,501,624		1,565,444
Performance stock units		17,415		121,907
Options to purchase common shares		16,671,003		16,148,020
Total potential shares excluded from diluted net loss per share		24,161,115		20,423,494

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12.

Leases

Finance Lease

The Company has a lease for a facility in Cranbury, New Jersey, consisting of 103,720 square feet of space including areas for offices, process development, research, and development laboratories and 50,000 square feet dedicated to AAV cGMP manufacturing facilities to support the Company’s pipeline (such lease, as amended, the “NJ Lease Agreement”). The NJ Lease Agreement has a 15-year term from September 1, 2019, with an option to renew for two consecutive five-year renewal terms.

Estimated rent payments for the NJ Lease Agreement are $1.2 million per annum, payable in monthly installments, and subject to annual base rent increases of 3%. The total commitment under the lease is estimated to be approximately $29.3 million over the 15-year term of the lease. The Company paid a cash security deposit of $0.3 million to the landlord in connection with the NJ Lease Agreement which has been reflected as part of deposits in the Consolidated Balance Sheets as of September 30, 2025, and December 31, 2024.

Operating Leases

On June 7, 2018, the Company entered into a three-year ESB Lease Agreement. In connection with the ESB Lease Agreement, the Company established an irrevocable standby letter of credit for $0.8 million. On March 26, 2021, the Company entered into Amendment No. 1 to the ESB Lease Agreement that extended the lease agreement to June 30, 2024. On March 29, 2024, the Company entered into Amendment No. 2 to the ESB Lease Agreement that extended the lease agreement to July 31, 2027. The letter of credit serves as the Company’s security deposit on the lease in which the landlord is the beneficiary and expires September 30, 2027.

The Company has a certificate of deposit of $0.8 million with a bank as collateral for the ESB Lease Agreement letter of credit which is classified as part of restricted cash in the Consolidated Balance Sheets as of September 30, 2025, and December 31, 2024.

On December 1, 2022, in connection with the acquisition of Renovacor, the Company added operating leases for space at facilities in Hopewell, New Jersey and Cambridge, Massachusetts with remaining lease terms of approximately 10.3 and 1.3 years, respectively. The Company recognized total right-of-use assets of $3.8 million with corresponding total lease liabilities of $3.6 million at lease commencement dates. The Company intends to sublease the facilities in Hopewell, New Jersey and signed the first agreement to sublease one of these facilities in January 2024. Rental income received under a sublease agreement was less than $0.1 million for the three and nine months ended September 30, 2025. Rental income received under a sublease agreement totaled $0.1 million and $0.3 million for the three and nine months ended September 30, 2024, respectively.

Rent expense excluding rental income was $0.3 million and $0.9 million for the three and nine months ended September 30, 2025. Rent expenses excluding rental income were $0.3 million and $1.2 million for the three and nine months ended September 30, 2024, respectively.

The total restricted cash balance for the Company’s operating and finance leases as of September 30, 2025, and December 31, 2024, was $0.8 million.

The following table summarizes lease cost for the nine months ended September 30, 2025, and 2024:


	Nine Months Ended September 30,
Lease cost	2025		2024
Operating lease cost	$	785	$	956
Finance lease cost:
Amortization of right of use assets		1,615		1,615
Interest on lease liabilities		1,418		1,413
Total lease cost	$	3,818	$	3,984

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The following table summarizes the future lease payments of the Company’s operating lease liabilities on an undiscounted cash flow basis:


Fiscal Year Ending December 31,	September 30, 2025
2025 (three months)	$	251
2026		1,005
2027		759
2028		522
2029		539
Thereafter		1,881
Total lease payments	$	4,957
Less: interest		(1,184	)
Total operating lease liabilities	$	3,773

The following table summarizes the future lease payments of the Company’s finance lease liabilities on an undiscounted cash flow basis:


Fiscal Year Ending December 31,	September 30, 2025
2025 (three months)	$	471
2026		1,911
2027		1,969
2028		2,028
2029		2,089
Thereafter		38,915
Total lease payments	$	47,383
Less: interest		(26,111	)
Total finance lease liability	$	21,272

The following table summarizes the operating and financing lease liabilities and right-of-use assets as of September 30, 2025, and December 31, 2024:


Leases	September 30, 2025		December 31, 2024
Operating right-of-use assets	$	3,653	$	4,173

Operating current lease liabilities	$	1,015	$	1,001
Operating noncurrent lease liabilities		2,758		3,258
Total operating lease liabilities	$	3,773	$	4,259

Finance right-of-use assets	$	40,748	$	42,363

Finance current lease liability	$	1,897	$	1,856
Finance noncurrent lease liability		19,375		19,383
Total finance lease liability	$	21,272	$	21,239


	Nine Months Ended September 30,
Other Information	2025			2024
Cash paid for amounts included in the measurement of lease liabilities:
Operating cash flows from operating leases	$	750		$	829
Cash flows from finance lease	$	1,385		$	1,333
Weighted-average remaining lease term - operating leases		6.2 years			6.7 years
Weighted-average remaining lease term - finance lease		18.9 years			19.9 years
Weighted-average discount rate - operating leases		8.83	%		8.82	%
Weighted-average discount rate - finance lease		8.96	%		8.96	%

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13.

Commitments and Contingencies

Litigation

In June 2025, the Company entered into a settlement agreement with Lexeo Therapeutics, Inc. (“Lexeo”) to resolve all claims in ongoing litigation between the parties in the United States District Court for the Southern District of New York. The litigation involved allegations by the Company of trade secret misappropriation and tortious interference, and counterclaims by Lexeo including correction of inventorship, breach of contract, and trade secret misappropriation. As part of a comprehensive resolution, Rocket also entered into separate settlement agreements on the same day with certain individuals formerly affiliated with the Company, who were also named in the litigation.

Under the terms of the settlement agreement between the Company and Lexeo, the litigation has been resolved amicably and fully, and without admission of liability by any party. For the three and nine months ended September 30, 2025, the gain or loss in connection with this settlement agreement was not material.

On June 11, 2025 and July 18, 2025, two stockholders filed putative securities class action lawsuits against us and certain of our executive officers in the United States District Court for the District of New Jersey, purportedly on behalf of classes of the Company’s investors who purchased or otherwise acquired the Company’s common stock between February 27, 2025 and May 26, 2025 and between September 17, 2024 and May 26, 2025, respectively. The complaints allege violations of Sections 10(b) and 20(a) of the Securities Exchange Act of 1934 and Rule 10b-5 promulgated thereunder in connection with various public statements made by the Company regarding its Phase 2 clinical trial for RP-A501 for Danon disease. The actions seek unspecified damages, costs and expenses, including attorneys’ fees. On September 9, 2025, the Court consolidated the two pending putative securities class action lawsuits, appointed two stockholders as co-lead plaintiffs, and approved their selection of co-lead counsel. Pursuant to a stipulation approved by the Court on September 22, 2025, the co-lead plaintiffs will have until November 18, 2025 to file a consolidated amended complaint. The Company intends to vigorously defend against such allegations. Given the nature of the cases, including that the proceedings are in their early stages, the Company is unable to predict the ultimate outcome of the cases or estimate the range of potential loss, if any.

On October 22, 2025, a putative derivative action was filed in the District of New Jersey, naming as Defendants certain of our officers and present or former directors of the Company. The Complaint (which names the Company as a nominal defendant) alleges that the Defendants engaged in wrongful conduct during the period from September 17, 2024 through May 26, 2025. The allegations in the complaint largely parallel the allegations made in the previously filed putative securities class action complaints, with some additional allegations regarding a supposed lack of internal controls and purported insider trading. The Complaint seeks declaratory relief, an award of damages to the Company, an order directing the Company and the individual defendants to institute certain requested corporate governance reforms, restitution from the individual defendants, and costs and disbursements related to the lawsuit. The Company intends to vigorously defend the litigation. Given the nature of the litigation, including the fact that the litigation is in its early stages, the Company is unable to predict the ultimate outcome of the litigation or estimate the range of potential loss, if any.

From time to time, the Company may be subject to various legal proceedings and claims that arise in the ordinary course of its business activities. Although the results of litigation and claims cannot be predicted with certainty, the Company does not believe it is party to any other claim or litigation the outcome of which, if determined adversely to the Company, would individually or in the aggregate be reasonably expected to have a material adverse effect on its business. Regardless of the outcome, litigation can have an adverse impact on the Company because of defense and settlement costs, diversion of management resources, and other factors.

Indemnification Arrangements

Pursuant to its bylaws and as permitted under Delaware law, the Company has indemnification obligations to directors, officers, employees or agents of the Company or anyone serving in these capacities. The maximum potential number of future payments the Company could be required to pay is unlimited. The Company has insurance that reduces its monetary exposure and would enable it to recover a portion of any future amounts paid. As a result, the Company believes that the estimated fair value of these indemnification commitments is minimal.

Throughout the normal course of business, the Company has agreements with vendors that provide goods and services required by the Company to run its business. In some instances, vendor agreements include language that requires the Company to indemnify the vendor from certain damages caused by the Company’s use of the vendor’s goods and/or services. The Company has insurance that would allow it to recover a portion of any future amounts that could arise from these indemnifications. As a result, the Company believes that the estimated fair value of these indemnification commitments is minimal.

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Contingent Consulting Fee

The Company has an agreement with a consultant that requires payment of a success fee of $0.3 million upon FDA approval of KRESLADI™ before January 31, 2026.

14.

Agreements Related to Intellectual Property

The Company, directly and through its subsidiary Spacecraft Seven, LLC, has various licenses and research and collaboration arrangements. The transactions principally resulted in the acquisition of rights to intellectual property that is in the preclinical phase and has not been tested for safety or feasibility. In all cases, the Company did not acquire tangible assets, processes, protocols, or operating systems. The Company expenses the acquired intellectual property rights as of the acquisition date on the basis that the cost of intangible assets purchased from others for use in R&D activities has no alternative future uses.

15.

CIRM Grants

LAD-I CIRM Grant

On April 30, 2019, CIRM awarded the Company up to $7.5 million under a CLIN2 grant to support the clinical development of its LV-based gene therapy, RP-L201. Proceeds from the grant helped fund clinical trial costs as well as manufactured drug product for Phase 1/2 patients enrolled at the U.S. clinical site, University of California, Los Angeles Mattel Children’s Hospital, led by principal investigator Donald Kohn, M.D., UCLA Professor of Microbiology, Immunology and Molecular Genetics, Pediatrics (Hematology/Oncology), Molecular and Medical Pharmacology and member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA. As of September 30, 2025, the Company has received $5.9 million in total RP-L201 grants from CIRM. The Company received a final milestone grant of less than $0.1 million on January 2, 2024, and no additional payments are available under the grant awards program.

DD CIRM Grant

On August 18, 2024, CIRM awarded the Company up to $5.8 million under a CLIN2 grant award to support the clinical development of its AAV-based gene therapy, RP-A501 for the treatment of DD. Proceeds from the grant would help fund clinical trial costs as well as manufactured drug product for Phase 1/2 patients. During the nine months ended September 30, 2025, the Company received grants of $2.7 million, which were recorded as a reduction of R&D expenses. Through September 30, 2025, the Company has received RP-A501 grants of $5.0 million from CIRM.

16.

Related Party Transactions

In June 2023, the Company entered into a consulting agreement with the spouse of one of the Company’s former executive officers for information technology advisory services. The Company incurred expenses of approximately $0 and $2,000 for the nine months ended September 30, 2025 and 2024, respectively, for services provided under this agreement.

In December 2024, in connection with a public offering, the Company sold 400,000 pre-funded warrants to purchase shares of the Company’s common stock to funds affiliated with RTW, the Company’s largest shareholder (see Note 8 “Stockholders’ Equity”).

In February 2025, the Company entered into a consulting agreement with one of the Company’s board members, effective March 3, 2025, for services related to the Company’s R&D activities. As compensation for services rendered during 2025, the consultant will receive $125,000 to be paid in equal monthly installments and $125,000 of RSU’s valued as of the closing price on March 3, 2025, which will cliff vest on December 31, 2025. The agreement will terminate on December 31, 2025, unless terminated earlier by the Company for cause or voluntarily by the consultant. The board member was paid approximately $87,500 for the nine months ended September 30, 2025, for services provided under the consulting agreement.

In June 2025, the Company received a total of approximately $215,000, representing return of short-swing profits from the sale of common stock by beneficial owners under Section 16(b) of the Securities and Exchange Act of 1934, as amended. The Company recognized these proceeds as a capital contribution and reflected a corresponding increase to additional paid-in capital.

In August 2025, the Company paid a total of approximately $67,000, representing payment of taxes upon vesting of RSUs by beneficial owners under Section 16(b) of the Securities and Exchange Act of 1934, as amended. The Company recognized these payments as capital payments and reflected a corresponding decrease to additional paid-in capital.

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17.

401(k) Savings Plan

The Company has a defined contribution savings plan (the “Plan”) under Section 401(k) of the Internal Revenue Code of 1986. This Plan covers substantially all employees who meet the minimum age and service requirements and allows participants to defer a portion of their annual compensation on a pre-tax basis. Company contributions to the Plan may be made at the discretion of the Company’s Board of Directors. The Company has elected the safe harbor match of 4% of employee contributions to the Plan, subject to certain limitations. The Company’s matching contribution for the three and nine months ended September 30, 2025, was $0.4 million and $1.5 million, respectively. The Company’s matching contribution for the three and nine months ended September 30, 2024, was $0.5 million and $1.3 million, respectively.

ROCKET PHARMACEUTICALS, INC. 10-Q